Alex's Lemonade Stand Foundation for Childhood Cancer

Neuroblastoma is a cancer that develops in young children and is one of the leading causes of pediatric cancer-related death. While many children can be cured, those with high-risk disease often relapse despite intensive therapy, including chemotherapy, radiation, surgery, and antibody treatments. For these patients, new and more effective treatment strategies are urgently needed.

Pediatric high-grade gliomas are among the deadliest childhood cancers. Survival has not improved in decades because these tumors grow quickly, resist standard treatments, and often return even after surgery, radiation, or chemotherapy. A small subset of these tumors carries an H3K27M mutation and shows high levels of a molecule called GD2 on the tumor surface. Early clinical trials using GD2-targeting CAR T cells, immune cells engineered to recognize GD2, have shown encouraging responses in these children. However, most high-grade gliomas do not carry this mutation.

Ewing sarcoma is a rare but deadly cancer that affects children and adolescents. It has a very poor prognosis and currently lacks effective treatment options. In recent years, a new type of therapy named “CAR T cells” has shown great results in several types of pediatric cancer.

Pediatric acute myeloid leukemia (AML) is a type of blood cancer affecting children and adolescents. Despite advances in therapy, survival has improved only modestly over the past decades, and relapse or treatment-resistant disease remains deadly. In AML, genetic changes can hijack the cell’s normal control systems, making key “switchboard operators” - proteins called transcription factors that coordinate multiple cellular programs - drive cells toward cancerous growth. Targeting these switchboard operators directly has proven extremely difficult.